P-012: FREQUENCY OF INDICATIONS OF HEMATOPOIETIC STEM CELL TRANSPLANT ACCORDING TO DIFFERENT SETS OF INDICATIONS AND ANALYSIS OF COMORBIDITY INDEX SCORES IN BRAZILIAN ADULTS WITH SICKLE CELL DISEASE
Authors: SANTOS G., TOZATTO-MAIO K., ALVES L., FONSECA G., MELO G., SUGANUMA-OTSUKA L., NOGUEIRA F., CARDOSO A., GUALANDRO S., ROCHA V.
Journal: HemaSphere (2022)

Purpose: Hematopoietic stem cell transplantation (HSCT) is the only curative therapy available for sickle cell disease (SCD). However, HSCT indications vary across countries, and HSCT mortality scores lack validation in SCD settings. Objectives: To identify how many adult patients followed at the SCD outpatient service of the Hospital das Clínicas de São Paulo meet at least one indication for HSCT, according to the Brazilian Ministry of Health (BMH) or to an international expert panel (Angelucci et al, 2014); to characterize the distribution of the hematopoietic cell transplantation comorbidity index (HCT-CI) and Charlson comorbidity index (CCI) scores, comparing them between transplantable and non-transplantable population to correlate with the severity of the disease. Materials and methods: Single-center, retrospective study. Using the set of HSCT indications from the Brazilian Ministry of Health (BMH) or Angelucci et al, we determined the frequency of patients with >1 indication. The concordance between the two sets was assessed using Cohen’s kappa coefficient. HCT-CI and CCI were used to assess risk of HSCT-related severity; median scores in transplantable and non-transplantable populations were compared using Mann-Whitney. Results: We included 219 SCD adults (143 SS,47 SC and 29 Sβ); 138 were female;the median age was 38 (range,20-74) years. 33 patients had ischemic stroke, 2 had hemorrhagic stroke and 10 Moya-moya. 125 patients had acute chest syndrome and 210 had vaso-occlusive crisis (VOC). 11 patients had pulmonary arterial hypertension,73 avascular necrosis,35 priapism,65 proliferative retinopathy and 184 proteinuria. 103 patients were under hydroxyurea. 186 received blood transfusion; 72 were alloimmunized. 168 patients(77%)had at least one HSCT indication, 108(50%) by BMH and 166(76%) by Angelucci;60 met an indication only by Angelucci and 2 only by BMH.The overall agreement rate between the 2 sets was 0.71,with a Cohen’s kappa coefficient of 0.44. Considering only SS and Sβ0 patients,the Cohen’s kappa remains moderate (0.5). The main indication by both sets was alloimmunization,followed by CNS event(BMH) and recurrent VOC(Angelucci). Among age groups, the percentage of patients with >1 TCTH indication was:18-30 years:70%;31-40 years:85%; 41-50 years:71%;51-60 years:77%;+60 years:90%. Median HCT-CI in the transplantable population was 2(range,0-12) and 1(range,0-5) in the non-transplantable(p=0.004);median CCI was respectively 1(range,1-9) and 0(range,0-3)(p<0.001). Conclusion: HSCT indications according to the BMH are stricter than those by Angelucci, explaining the moderate agreement on Cohen’s kappa coefficient. The rate of indications is higher than previously reported due to the higher rate of alloimmunization and older median age of our cohort, as HSCT indications increase over time. Median HCT-CI and CCI were significantly higher in the population with >1 HSCT indication, reflecting its higher severity. This study shows that indication of HSCT should be counterbalanced with the risk of mortality of SCD and HSCT. Dynamic scores of mortality should be further validated to predict the best moment for HSCT in adults with SCD. The authors do not declare any conflict of interest